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Sickle Cell Crispr

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Last Updated: 22 September 2020

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General | Latest Info

Sickle cell disease is a complex disease that affects the structure and function of hemoglobin, molecule in red blood cells that delivers oxygen around the body. Disease is caused by a single mutation in - globin GENE that results in malformed hemoglobin, which distorts red blood cells into a hard, sickle shape, and leads to a range of diverse clinical manifestations such as blockage of blood vessels, debilitating and frequent pain crises, chronic organ damage, poor quality of life, and early death. According to a study published in 2017 in New England Journal of Medicine, sickle cell disease is one of the most common inherited blood disorders, affecting approximately 90 000 people in the United States and hundreds of thousands of people worldwide. The first evidence of sickle cell disease surfaced over a century ago when physicians analyzed blood of affected patients and reported peculiar elongated and sickle - sharped cells. It wasnt until 1949, in a report published in Journal Science by Linus Pauling and his colleagues, that link between abnormality in red blood cell shape with mutations in patients ' amino acid sequences was make; this was the first time genetic disease was linked to protein mutation. While many advances have been made that have greatly improved the lives of patients with sickle cell disease, available treatments are limited and confined to minimizing symptoms. Hematopoietic stem cell transplantation is only a curative, nonexperimental option FOR patients with severe forms of disease. However, despite the high success rate, suitable donors are rare and there exists a risk of graft rejection and potentially fatal graft - versus - host disease.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions

The journey

Frangoul say they use medication to mobilize stem cells from bone marrow and push them into blood. Then researchers can collect Stem Cells. Cells get shipped to lab to undergo Gene Editing, then theyre frozen. In meantime, Gray was getting blood transfusions to keep her condition under control. After a round of chemotherapy, Gene - Edit Cells are infused back into the body. Prior to her treatment, Gray averaged seven hospitalizations a year and had multiple blood transfusions. Her hemoglobin was 7 and she had little energy. She has not been hospitalized and her hemoglobin is 11, so she has more energy. She been able to spend time with her family. She is doing really well, Frangoul say. I am extremely excited about her progress.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions

Improved tools for genome editing

Potential gene therapy - base treatment for sickle Cell Disease patients. In theory, both Hematopoietic Stem cells and induced pluripotent Stem cells can be used for therapy. In ideal case, HSCs isolated from patients can be corrected for mutation in vitro, followed by transplantation into patient. As an alternative, somatic cells such as skin or blood cells from patients can first be reprogrammed into iPSCs, followed by correction of cells and differentiation into HSCs. These correct HSCs can then be transplanted back into patients to create healthy blood cells. Procedure for iPSCs is indicated with dashed arrows. Even though both strategies are theoretically viable, there are various issues to be addrest before use of HSCs or iPSCs for therapy. For further discussion, please see section 4.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions

The path forward

What will it take for CRISPR babies to become medically acceptable? Earlier this month, international commission of scientists released a highly anticipated report detailing steps needed to turn the gene - editing fiasco into a powerful treatment that could wipe out genetic diseases throughout generations. Conclusion: Editing genome of human embryos to make CRISPR babies isnt yet safe. But as technology matures, it could be, and there will be countries that greenlight procedure. First sanction CRISPR baby experiments should be narrowly restricted to couples who otherwise cannot have healthy children relatively rare pool of people likely to require international, collaborative supervision. The report stands out in that it doesnt explicitly discuss moral and ethical issues relating to CRISPR babies. It also does weigh in on the merits of technology or whether there should be a global moratorium. Rather, report envisions a world where CRISPR babies will eventually be allow, and digs into the nitty - gritty of technical and oversight requirements for the procedure to become medically acceptable. As Dr. Richard Lifton, co - chair of the commission say, report acts as manual for stag rollout of CRISPR babies, with a focus on safety first and foremost. One recommendation that especially stands out was call for a whistleblower system to potentially nip unsanctioned CRISPR baby experiments in bud. There will always be rogue scientists, says co - chair Dr. Kay Davies. Going forward, international cooperation and open discussion of all aspects of Genome Editing will be essential.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions

Signs of progress

In both cases, transplant cells flourish in the bone marrow of recipients, without any serious safety concerns, but do not produce clear medical benefit. In men treated for HIV, researchers attempted to use CRISPR to disable protein that many strains of HIV use to enter cells. But only 5% of transplanted cells were edited not enough to cure disease, researchers reported on September 1. Study has been place on hold while researchers explore ways to boost that percentage, said Hongkui Deng, stem - cell researcher at Peking University in Beijing and lead author of the work. There are early hints that another trial might meet with more success. Crispr Therapeutics in Cambridge, Massachusetts, and Vertex Pharmaceuticals in Boston, Massachusetts, have treated two people with genetic disorders sickle - cell anaemia and - thalassaemia. Both deplete oxygen - carrying haemoglobin molecules in the blood:. The idea is to use CRISPR to disable gene that otherwise shut off production of another form of haemoglobin. Early results suggest that treatment might have eased some symptoms of disorders, but participants will need to be followed for a longer period to be sure. Other researchers are already itching to move beyond editing cells in dish. The challenge is in finding ways to transport gene - editing machinery to where it is needed in the body, say John Leonard, chief executive of Intellia Therapeutics, biotechnology company in Cambridge, Massachusetts, that is focus on CRISPR - Cas9 genome Editing. Delivery approach is so important. Last July, pharmaceutical companies Editas Medicine in Cambridge, Massachusetts, and Allergan in Dublin launched a trial to treat the genetic disorder Leber congenital amaurosis 10, which can cause blindness, by editing eye cells. Researchers will inject into the eye virus containing DNA that encodes CRISPR genome - Editing machinery, bypassing need to guide those tools through bloodstream to specific tissues. Viruss will be responsible for carrying genome - editing tools into cells. It is the first trial to attempt CRISPR - Cas9 gene Editing inside the body, and early results could be reported this year. That would be a landmark moment for the field, and could pave the way for future trials targeting other organs, said Charles Gersbach, bioengineer at Duke University in Durham, North Carolina. But he and others say that they hope researchers will eventually move away from using viruses to shuttle genome - Editing machinery into cells. Deactivate viruses can still sometimes provoke immune responses, and can only carry a limited amount of DNA.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions

Shrink to fit

Doctors in the US have used the gene - editing tool CRISPR to treat patients with genetic disorder for the very first time. But researchers caution that the procedure done as part of an ongoing trial for sickle cell Disease is still experimental and that its use must be weighed against other treatment options. Sickle cell Disease affects up to 100 000 Americans and is most common in those of African descent. According to the Centers for Disease Control and Prevention, genetic defect in patients ' hemoglobin, blood protein that carries oxygen, causes cells to become hard and sticky, and take on an abnormal sickle shape that can cause blockages and lead to stroke and organ damage, among other complications. Currently, the only cure for sickle cell disease is bone marrow or stem cell transplant, but many patients are unable to find a suitable donor. With the CRISPR technique, which involves using enzyme to edit DNA at a precise location, scientists can alter patients ' own blood cells to make them produce hemoglobin variant normally only made by fetuses and babies for a short time after birth. Following chemotherapy to wipe out any existing defective cells, new cells can then be returned to patient in hopes that theyll treat disease. Though technique has shown promise in earlier primate studies, according to results published last month in the journal Science Translational Medicine, for 34 - year - old Victoria Gray, first human volunteer, it will likely take months to determine whether altered cells are performing as predict. Having lived with pain from sickle cell Disease throughout her life, Gray told NPR treatment gives me hope if it gives me nothing else. The trial is being jointly conducted by Switzerland - Base CRISPR Therapeutics and Vertex Pharmaceuticals, which is headquarter in Boston, and will ultimately enroll up to 45 participants. While other pharmaceutical companies are taking varying approaches to treating disease, including different gene therapies, high prices remain an issue. The CRISPR approach, researchers say, requires fewer cells and therefore could be more cost - effective. Human trials of CRISPR have ramped up this year. Doctors at the University of Pennsylvania are trying techniques to treat cancer patients, while Massachusetts Eye and Ear Infirmary in Boston is currently recruiting for studies to edit cells in the retinas of patients with inherited blindness. Still, US approval can be slow - moving compared to other countries. Many more cancer trials are underway in China, though use of CRISPR to edit genomes of twin girls there last year caused international outcry and led the World Health Organization last week to call for any experiments that would lead to more gene - edited babies to be halt. Doctors at the University of California in San Francisco have found a way to successfully extract people's speech directly from their brain, according to a study published on Tuesday in the journal Nature Communications.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions

Sources

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions.

* Please keep in mind that all text is machine-generated, we do not bear any responsibility, and you should always get advice from professionals before taking any actions

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